索拉非尼治疗进展期胆管细胞型肝癌的临床观察

Clinical observation of sorafenib for advanced cholangiocellular carcinoma

目的：观察索拉非尼治疗进展期胆管细胞型肝癌（CCC）的临床疗效及毒性反应。方法：15例进展期CCC患者口服索拉非尼400 mg,2次/d,3周为一个周期,持续用药,直至病情进展或出现不可耐受的毒性反应。观察服药12周时的有效率（RR）和疾病控制率（DCR）,次要观察指标包括至疾病进展时间（TTP）,无进展生存期（PFS）,总生存期（OS）,治疗持续时间（DOT）和毒性反应。结果：15例患者中位DOT是3.2（1.5-30）个月;按照RECIST标准评价部分缓解4例,病情稳定7例,疾病进展4例;12周时RR为26.7%,DCR为73.3%;中位TTP,PFS及OS分别为3.2、5.5和5.7个月;治疗期间出现不良反应者9例,最常见的毒副反应是1-2级皮疹（33.3%）,出现3级手足综合征1例（6.7%）。结论：索拉非尼对进展期CCC患者治疗有效,其不良反应可以耐受。

Objective： To observe the clinical efficacy and toxic effect of sorafenib in treatment of advanced cholangiocellular carcinoma（CCC）.Methods： Fifteen patients with advanced CCC were enrolled and underwent continuous oral sorafenib administration at a dose of 400 mg twice daily with 3 weeks as a treatment cycle until disease progression or intolerant drug-related toxicity. The primary endpoints included response rate（RR） and the disease control rate（DCR） at 12 weeks, and the secondary endpoints included time to progression（TTP）, progression-free survival（PFS）, overall survival（OS）, duration of treatment（DOT） and toxicity.Results： In the 15 patients, the median DOT was 3.2（1.5-30） months, 4 cases achieved a partial response and 7 cases obtained a stable disease, according to RECIST criteria. The median TTP, PFS and OS was 3.2, 5.5 and5.7 months, respectively. Treatment-related adverse reactions occurred in 9 cases, in which the grade 1-2 skin rash was most common toxicity reaction（33.3%）, and grade 3 hand-foot syndrome occurred in one case（6.7%）.Conclusion： Sorafenib monotherapy has proven efficacy and manageable toxicity in advanced CCC.